Overview of Adeno-Associated Viral Vectors

The use of adeno-associated virus (AAV) as a gene transfer vector has been steadily increasing over the past several years. A AV vectors have been particularly useful for applications where sustained gene expression is required. Prolonged in vivo expression following A AV treatment has been seen in the liver (1 ,2 ), brain (3 ,4 ), skeletal muscle (5 ,6 ), lung (7 ,8 ), and hematopoietic stem cells (9 ,10 ) of animal models. Therapeutic benefit from A AV treatment has been shown in a number of preclinical models of disease, including animal models of coagulopathies (11 ,12 ), lysosomal storage diseases (13 ,14 ), vision defects (15 ,16 ), and amino acid disorders (17 ). Clinical trials using A AV for the treatment of hemophilia B have begun, and early reports from these trials have been promising (18 ). In this introductory chapter to AAV, we will provide a brief overview of the molecular biology of this virus, an overview of methods of vector production, and a brief summary of the use of alternate AAV serotypes. The following chapters will then focus on specific methods and techniques for AAV transduction of the organs listed previously.